Safety and Efficacy Study of Intracystic TARA-002 for the Treatment of Lymphatic Malformations in Participants 6 Months to Less Than 18 Years of Age (STARBORN-1)

May 14, 2026 updated by: Protara Therapeutics

A Phase 2a/b Single Arm Open Label Study to Evaluate the Safety and Efficacy of Intracystic Administration of TARA-002 in Participants Between 6 Months to Less Than 18 Years of Age for the Treatment of Macrocystic and Mixed Cystic Lymphatic Malformations

This is a Phase 2a/b single arm open label study to evaluate the safety, reactogenicity, and efficacy of intracystic injection of TARA-002 in participants 6 months to less than 18 years of age for the treatment of macrocystic and mixed cystic lymphatic malformations. The Phase 2a safety lead-in, age de-escalation study is designed to establish the safety of TARA-002 in older participants 6 years to less than 18 years before proceeding to younger participants 2 years to less than 6 years, then 6 months to less than 2 years. The Phase 2b is an expansion study in which enrollment of participants will be initiated after safety has been established in each cohort during the Phase 2a safety lead-in study. Each participant will receive up to 4 injections of TARA-002 spaced approximately 6 weeks apart.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

38

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
    • Colorado
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
    • Florida
      • Jacksonville, Florida, United States, 32207
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Withdrawn
        • Cincinnati Children's Hospital Medical Center
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health & Science University
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Withdrawn
        • Children's Hospital of Philadelphia: Comprehensive Vascular Anomalies Program
    • Tennessee
      • Nashville, Tennessee, United States, 37232
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Recruiting
        • Children's Hospital of The King's Daughters
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male or female participants 6 months to less than 18 years of age at the time of informed consent/assent form was signed
  • Participants whose parent/LAR(s) have voluntarily given written consent and participants who provided assent (if applicable) after the study has been explained to them
  • Participants with macrocystic LM or mixed cystic LM (≥ 50% macrocystic disease measured by volume) of the Head/Neck/Mediastinum according to the ISSVA 2018 criteria (ISSVA 2018) measured via LM imaging at Screening to confirm, upon central review, the diagnosis of macrocystic or mixed cystic LM
  • Participants who may have had surgical or sclerotherapy treatment for their LM, but not within six months of the consent/assent form being signed

Exclusion Criteria:

  • Penicillin allergy
  • Vascular tumors or combined vascular malformations
  • Microcystic LM or mixed cystic LM with predominant microcystic features
  • LMs of the orbit (orbital LM) as target cyst

For more information on eligibility criteria, please contact the sponsor.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: TARA-002
TARA-002 is a lyophilized biological preparation for injection containing cells of Streptococcus pyogenes (Group A, type 3) Su strain treated with benzylpenicillin.
Biological: TARA-002
All participants will receive up to 4 intracystic injections spaced approximately 6 weeks apart.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Proportion of participants with clinical success after one treatment cycle of TARA-002
Time Frame: 8 weeks after the last injection
8 weeks after the last injection

Secondary Outcome Measures

Outcome Measure
Time Frame
Safety: Percentage of participants with solicited local site and systemic reactions
Time Frame: 14 days (2 weeks) after each injection
14 days (2 weeks) after each injection
Safety: Percentage of participants with unsolicited adverse events (AEs)
Time Frame: Through study completion (approximately 32 weeks after last injection)
Through study completion (approximately 32 weeks after last injection)
Safety: Percentage of participants with SAEs, AEs of special interest, AEs leading to premature discontinuation of study intervention, AEs leading to withdrawal from study, AEs with an outcome of death, and MAAEs
Time Frame: Through study completion (approximately 32 weeks after last injection)
Through study completion (approximately 32 weeks after last injection)
Durable Response: Proportion of participants with clinical success after one treatment cycle of TARA-002 assessed at 8 weeks after the last injection and maintained clinical success at 32 weeks after the last injection
Time Frame: 32 weeks after the last injection
32 weeks after the last injection
Clinical Success by LM Type: Proportion of participants with macrocystic LM or mixed cystic LM with clinical success after one treatment cycle of TARA-002
Time Frame: 8 weeks after the last injection
8 weeks after the last injection
Investigator Assessment: Proportion of participants who demonstrate clinical improvement, as assessed by the Investigator, after one treatment cycle of TARA-002 compared to baseline
Time Frame: 8 weeks after last injection and 32 weeks after last injection
8 weeks after last injection and 32 weeks after last injection
Quality of Life: Change in Quality-of-Life assessment based on Pediatric Quality of Life Inventory (PedsQL) and Visual Analog Scale (VAS) for Pain after one treatment cycle of TARA-002
Time Frame: 8 weeks after last injection and 32 weeks after last injection
8 weeks after last injection and 32 weeks after last injection

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Protara Therapeutics

Investigators

  • Study Director: Chief Scientific Operations Officer, Protara Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 18, 2023

Primary Completion (Estimated)

December 1, 2026

Study Completion (Estimated)

May 1, 2027

Study Registration Dates

First Submitted

May 11, 2023

First Submitted That Met QC Criteria

May 22, 2023

First Posted (Actual)

May 23, 2023

Study Record Updates

Last Update Posted (Actual)

May 18, 2026

Last Update Submitted That Met QC Criteria

May 14, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • TARA-002-201

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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